| RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model SQ Harper, PD Staber, X He, SL Eliason, IH Martins, Q Mao, L Yang, ... Proceedings of the National Academy of Sciences 102 (16), 5820-5825, 2005 | 850 | 2005 |
| RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia H Xia, Q Mao, SL Eliason, SQ Harper, IH Martins, HT Orr, HL Paulson, ... Nature medicine 10 (8), 816-820, 2004 | 824 | 2004 |
| The bifunctional microRNA miR-9/miR-9* regulates REST and CoREST and is downregulated in Huntington's disease AN Packer, Y Xing, SQ Harper, L Jones, BL Davidson Journal of Neuroscience 28 (53), 14341-14346, 2008 | 800 | 2008 |
| Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi JL McBride, RL Boudreau, SQ Harper, PD Staber, AM Monteys, I Martins, ... Proceedings of the National Academy of Sciences 105 (15), 5868-5873, 2008 | 714 | 2008 |
| Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy SQ Harper, MA Hauser, C DelloRusso, D Duan, RW Crawford, SF Phelps, ... Nature medicine 8 (3), 253-261, 2002 | 678 | 2002 |
| Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6 MJ Blankinship, P Gregorevic, JM Allen, SQ Harper, H Harper, CL Halbert, ... Molecular therapy 10 (4), 671-678, 2004 | 312 | 2004 |
| CHIP suppresses polyglutamine aggregation and toxicity in vitro and in vivo VM Miller, RF Nelson, CM Gouvion, A Williams, E Rodriguez-Lebron, ... Journal of Neuroscience 25 (40), 9152-9161, 2005 | 300 | 2005 |
| DUX4, a candidate gene for facioscapulohumeral muscular dystrophy, causes p53‐dependent myopathy in vivo LM Wallace, SE Garwick, W Mei, A Belayew, F Coppee, KJ Ladner, ... Annals of neurology 69 (3), 540-552, 2011 | 271 | 2011 |
| Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury M Liu, Y Yue, SQ Harper, RW Grange, JS Chamberlain, D Duan Molecular Therapy 11 (2), 245-256, 2005 | 241 | 2005 |
| Microdystrophin Gene Therapy of Cardiomyopathy Restores Dystrophin-Glycoprotein Complex and Improves Sarcolemma Integrity in the Mdx Mouse Heart Y Yue, Z Li, SQ Harper, RL Davisson, JS Chamberlain, D Duan Circulation 108 (13), 1626-1632, 2003 | 181 | 2003 |
| Nucleic acid silencing of Huntington's Disease gene B Davidson, S Harper US Patent App. 11/048,627, 2005 | 132 | 2005 |
| RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy LM Wallace, J Liu, JS Domire, SE Garwick-Coppens, SM Guckes, ... Molecular Therapy 20 (7), 1417-1423, 2012 | 118 | 2012 |
| Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin JM Scott, S Li, SQ Harper, R Welikson, D Bourque, C DelloRusso, ... Neuromuscular Disorders 12, S23-S29, 2002 | 116 | 2002 |
| RNA interference suppression of neurodegenerative diseases and methods of use thereof BL Davidson, H Xia, Q Mao, H Paulson, R Boudreau, S Harper US Patent 8,481,710, 2013 | 78 | 2013 |
| Connecdenn, a novel DENN domain-containing protein of neuronal clathrin-coated vesicles functioning in synaptic vesicle endocytosis PD Allaire, B Ritter, S Thomas, JL Burman, AY Denisov, ... Journal of Neuroscience 26 (51), 13202-13212, 2006 | 78 | 2006 |
| Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD) E Ansseau, C Vanderplanck, A Wauters, SQ Harper, F Coppée, ... Genes 8 (3), 93, 2017 | 66 | 2017 |
| AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD CR Giesige, LM Wallace, KN Heller, JO Eidahl, NY Saad, AM Fowler, ... JCI insight 3 (22), 2018 | 64 | 2018 |
| Progress and challenges in RNA interference therapy for Huntington disease SQ Harper Archives of neurology 66 (8), 933-938, 2009 | 63 | 2009 |
| Pre-clinical safety and off-target studies to support translation of AAV-mediated RNAi therapy for FSHD LM Wallace, NY Saad, NK Pyne, AM Fowler, JO Eidahl, JS Domire, ... Molecular therapy Methods & clinical development 8, 121-130, 2018 | 57 | 2018 |
| Conditional over-expression of PITX1 causes skeletal muscle dystrophy in mice SN Pandey, J Cabotage, R Shi, M Dixit, M Sutherland, J Liu, S Muger, ... Biology open 1 (7), 629-639, 2012 | 57 | 2012 |
